Teaching hospital
Appears in 3 stories
Lead sponsor of the RAG-18 Phase I trial
Three boys with Duchenne muscular dystrophy received monthly infusions of an experimental drug. Four months later, muscle biopsies showed 3.5- to 5.3-fold increases in utrophin, a protein that can stand in for the missing dystrophin.
Updated 7 days ago
Lead trial site for the China Phase I/III study
About 1.5 million people worldwide have retinitis pigmentosa, an inherited disease that destroys the retina's light-sensing cells and can end in blindness. The only approved treatment covers patients with a specific RPE65 gene mutation — a small fraction of the affected population.
Updated Sep 29
Lead site for GENESIS-IPF-3
Insilico Medicine dosed the first patient this week in a Phase III trial of Rentosertib, a drug for the progressive lung disease idiopathic pulmonary fibrosis. It's the first medicine with both an AI-identified target and an AI-designed molecule to reach this stage of testing.
Updated Sep 11
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